A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…
Two years of treatment with the experimental enzyme replacement therapy PRX-102 (pegunigalsidase alfa) prevented kidney decline in adults with Fabry disease and showed safety and effectiveness similar to Fabrazyme (agalsidase beta), top-line data from the BALANCE Phase 3 clinical trial show. In addition to meeting the trial’s primary goal…
People with classic Fabry disease due to a common disease-causing mutation who don’t smoke can still have poorer lung health and symptoms like dry cough and shortness of breath, but these symptoms can stabilize with enzyme replacement therapy (ERT), a small study in Finland suggests. These findings, in a group…
The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…
The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…
Members of the Fabry disease community are invited to share their stories — those of patients, caregivers, family members, and advocates — and to use social media to celebrate Fabry Awareness Month this April. For this year’s campaign, the global Fabry International Network, known as FIN, has launched…
JCR Pharmaceuticals and Sumitomo Dainippon Pharma have teamed up to market and commercialize in Japan agalsidase beta BS, a biosimilar of Fabrazyme, an already approved treatment designed to replace a critical enzyme missing in people with Fabry disease. According to a company press release, “This…
The next patient to receive FLT190, an investigational gene therapy for Fabry disease, in the ongoing Phase 1/2 MARVEL-1 trial will be given a second, higher dose of therapy, accelerating an earlier trial timeline. This person was originally intended to be included in the first, lowest dose group of…
Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…
Updated findings in an ongoing Phase 1/2 clinical trial of Sangamo Therapeutics‘ gene therapy ST-920 (isaralgagene civaparvovec) shows the treatment continues to be well tolerated and effective in adults with Fabry disease. As of the Nov. 19 data cutoff date, all five treated patients enrolled showed normalized levels of…
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